Nanotechnology Now

Our NanoNews Digest Sponsors

Heifer International

Wikipedia Affiliate Button

Home > Press > Alnylam and MIT Collaborators Publish Data on Novel Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics – New Pre-clinical Findings Demonstrate that Binary Combinations of Novel “Lipidoids” Result in Synergistic Effects on Target Gene Silencing –

Abstract:
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, together with collaborators at the Massachusetts Institute of Technology (MIT), announced today the publication of new data describing a novel approach for systemic delivery of RNAi therapeutics using synergistic combinations of novel lipid-like materials called "lipidoids."

Alnylam and MIT Collaborators Publish Data on Novel Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics – New Pre-clinical Findings Demonstrate that Binary Combinations of Novel “Lipidoids” Result in Synergistic Effects on Target Gene Silencing –

Cambridge, MA | Posted on July 19th, 2011

"These new data reflect our continued and broad-based commitment to developing novel approaches for the systemic delivery of RNAi therapeutics. In working with world-renowned academic leaders in the field of drug delivery at MIT, we continue to expand our understanding and knowledge about innovative delivery approaches," said Kevin Fitzgerald, Ph.D., Senior Director of Research at Alnylam. "These recently published data show that binary combinations of specific lipidoids can result in optimized lipid nanoparticle formulations with synergistic siRNA delivery properties."

"Our long-standing collaborative efforts with Alnylam continue to be very productive as evidenced by this current publication, marking the 12th peer-reviewed paper we have co-authored together," said Dan Anderson, Ph.D., of the David H. Koch Institute for Integrative Research at MIT. "As we continue in these joint efforts, we see boundless opportunities for novel delivery strategies for RNAi therapeutics and we look forward to continued progress."

The new research results, which were published in the journal Molecular Therapy (Whitehead et al., MolTher, 2011 July 12, doi:10.1038/mt.2011.141), established that binary combinations of lipidoids can be formulated together in a single lipid nanoparticle (LNP) formulation to achieve synergistic gene silencing effects both in vitro and in vivo. In the current study, more than 3,500 novel LNP formulations were prepared and then evaluated for effects on gene silencing. Results demonstrated achievement of synergistic gene silencing when utilizing materials that, when combined, mediated both efficient cellular uptake and productive endosomal escape. The pre-clinical data showed that ineffective single lipidoids could be formulated together to induce robust silencing of the luciferase mRNA in vitro and the Factor VII mRNA in vivo. Importantly, it is anticipated that this binary formulation strategy could be applicable to any siRNA delivery material in any target cell population that utilizes combinations of unique components to mediate distinct steps in the delivery process, for example lipids, polymers, and siRNA conjugates.

About RNA Interference (RNAi)

RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.

####

About Alnylam Pharmaceuticals, Inc.
Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, and ALN-HPN for the treatment of refractory anemia. As part of its “Alnylam 5x15TM” strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington’s disease. The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and Sanofi. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world’s top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts.

Alnylam Forward-Looking Statements

Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including without limitation, statements regarding Alnylam’s expectations with respect to its “Alnylam 5x15” product strategy, and Alnylam’s views with respect to the potential for systemic delivery of RNAi therapeutics using synergistic combinations of novel lipid-like materials called “lipidoids,” constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Alnylam’s ability to discover and develop novel delivery approaches and novel drug candidates, successfully demonstrate the efficacy and safety of its drug candidates in human clinical trials and establish and maintain strategic business alliances and new business initiatives, as well as those risks more fully discussed in the “Risk Factors” section of its most recent annual report on Form 10-Q on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam does not assume any obligation to update any forward-looking statements.

For more information, please click here

Contacts:
Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Senior Director, Investor Relations and
Corporate Communications
or
Media:
Spectrum
Amanda Sellers, 202-955-6222 x2597

Copyright © Business Wire

If you have a comment, please Contact us.

Issuers of news releases, not 7th Wave, Inc. or Nanotechnology Now, are solely responsible for the accuracy of the content.

Bookmark:
Delicious Digg Newsvine Google Yahoo Reddit Magnoliacom Furl Facebook

Related News Press

News and information

Eric Berger Wins the National Space Society's 2017 Space Pioneer Award for Mass Media January 19th, 2017

Nanometrics to Announce Fourth Quarter and Full Year Financial Results on February 7, 2017 January 19th, 2017

'5-D protein fingerprinting' could give insights into Alzheimer's, Parkinson's January 19th, 2017

Strength of hair inspires new materials for body armor January 18th, 2017

Nanomedicine

'5-D protein fingerprinting' could give insights into Alzheimer's, Parkinson's January 19th, 2017

New active filaments mimic biology to transport nano-cargo: A new design for a fully biocompatible motility engine transports colloidal particles faster than diffusion with active filaments January 11th, 2017

Keystone Nano Announces FDA Approval Of Investigational New Drug Application For Ceramide NanoLiposome For The Improved Treatment Of Cancer January 10th, 2017

Captured on video: DNA nanotubes build a bridge between 2 molecular posts: Research may lead to new lines of direct communication with cells January 9th, 2017

Discoveries

'5-D protein fingerprinting' could give insights into Alzheimer's, Parkinson's January 19th, 2017

Strength of hair inspires new materials for body armor January 18th, 2017

Self-assembling particles brighten future of LED lighting January 18th, 2017

Dressing a metal in various colors: DGIST research developed a technology to coat metal with several nanometers of semiconducting materials January 17th, 2017

Announcements

Eric Berger Wins the National Space Society's 2017 Space Pioneer Award for Mass Media January 19th, 2017

Nanometrics to Announce Fourth Quarter and Full Year Financial Results on February 7, 2017 January 19th, 2017

'5-D protein fingerprinting' could give insights into Alzheimer's, Parkinson's January 19th, 2017

Strength of hair inspires new materials for body armor January 18th, 2017

Nanobiotechnology

'5-D protein fingerprinting' could give insights into Alzheimer's, Parkinson's January 19th, 2017

Nanoscale Modifications can be used to Engineer Electrical Contacts for Nanodevices January 13th, 2017

New active filaments mimic biology to transport nano-cargo: A new design for a fully biocompatible motility engine transports colloidal particles faster than diffusion with active filaments January 11th, 2017

Keystone Nano Announces FDA Approval Of Investigational New Drug Application For Ceramide NanoLiposome For The Improved Treatment Of Cancer January 10th, 2017

Alliances/Trade associations/Partnerships/Distributorships

GLOBALFOUNDRIES Expands Partner Program to Speed Time-to-Market of FDX™ Solutions: Increased support affirms FDXcelerator™ Program’s vital role in promoting broader deployment of GLOBALFOUNDRIES’ FDX™ portfolio December 15th, 2016

Infrared instrumentation leader secures exclusive use of Vantablack coating December 5th, 2016

Leti and Grenoble Partners Demonstrate World’s 1st Qubit Device Fabricated in CMOS Process: Paper by Leti, Inac and University of Grenoble Alpes Published in Nature Communications November 28th, 2016

Mechanism for sodium storage in 2-D material: Tin selenide is an effective host for storing sodium ions, making it a promising material for sodium ion batteries October 27th, 2016

Research partnerships

Chemistry on the edge: Experiments at Berkeley Lab confirm that structural defects at the periphery are key in catalyst function January 13th, 2017

Recreating conditions inside stars with compact lasers: Scientists offer a new path to creating the extreme conditions found in stars, using ultra-short laser pulses irradiating nanowires January 12th, 2017

Zeroing in on the true nature of fluids within nanocapillaries: While exploring the behavior of fluids at the nanoscale, a group of researchers at the French National Center for Scientific Research discovered a peculiar state of fluid mixtures contained in microscopic channels January 11th, 2017

New active filaments mimic biology to transport nano-cargo: A new design for a fully biocompatible motility engine transports colloidal particles faster than diffusion with active filaments January 11th, 2017

NanoNews-Digest
The latest news from around the world, FREE




  Premium Products
NanoNews-Custom
Only the news you want to read!
 Learn More
NanoTech-Transfer
University Technology Transfer & Patents
 Learn More
NanoStrategies
Full-service, expert consulting
 Learn More











ASP
Nanotechnology Now Featured Books




NNN

The Hunger Project